Retinitis PigmentosaPhase 1/2RPNCT04315025
Safety Issues of Peribulbar Injection of UC-MSC in Patients With Retinitis Pigmentosa
A Phase 1/2 study testing Umbilical Cord Mesenchymal Stem Cell (UC-MSC) and Conditioned Medium (CM) in people with Retinitis Pigmentosa, run by PT. Prodia Stem Cell Indonesia at 2 sites in Indonesia.
Trial summary
The study will perform UC-MSCs and CM transplantation. The first group will be injected by UCMSC+NaCl. the 2nd group will be injected by UC-MSC+CM. the 3rd group will be injected by CM. Each group consists of 6 subjects. all groups will be transplanted via peribulbar route. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Retinitis Pigmentosa
- What is being tested?
- Umbilical Cord Mesenchymal Stem Cell (UC-MSC) and Conditioned Medium (CM)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesIndonesia
- Study dates
- 7 Oct 2018to 20 Sep 2019
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Visual field defects at initial examination with Humhprey perimetry are between 25% to 50%
- Willing to sign informed consent as research subjects
- Willing to do peribulbar injection with mesenchymal stem cells isolated from umbilical cord tissue
- Willing to do visual field checks with Humphrey's perimetry, vision tests with Snellen boards, Optical Coherrent Tomography (OCT) examinations, electroretinogram examinations and fill out a quality of life questionnaire
Who cannot join
- Pregnant or nursing women
- Positive result of HIV test
- Have a history of eye tumors
- In immunosuppressive treatment or other drugs that can affect the growth of transplanted stem cells
- Have another eye disease such as diabetic retinopathy, uveitis, cataract, and glaucoma
- Do not come to control according to the schedule determined by the researcher (loss to follow up)
Trial locations and contacts
2 sites in 1 country.
Indonesia2 sites
Jakarta Eye Center Hospital
Jakarta, DKI Jakarta
Sardjito Hospital
Yogyakarta, Special Region, 55284
About this study
Official title: Safety Issues of Peribulbar Injection of Umbilical Cord Mesenchymal Stem Cell (UC-MSC) in Patients With Retinitis Pigmentosa
The study will perform UC-MSCs and CM transplantation. The first group will be injected by UCMSC+NaCl. the 2nd group will be injected by UC-MSC+CM. the 3rd group will be injected by CM. Each group consists of 6 subjects. all groups will be transplanted via peribulbar route. the dosage of UC-MSC is 1 million cells for each subject. All groups will be observed until 6 months.
Read the detailed description
The eyes which will give the transplant should be given an aseptic and antiseptic technique to prevent the contamination from the inside and outside. Sterile cover attached to other parts of the face except for the eyes. 1.8 ml cell preparations are suspended in physiological NaCl until it reaches a total of 2 ml volume of cell suspension (for UC-MSC + NaCl group). Stem cell suspension will be injected by peribulbar and if the injection was done, patients will be given a quinolone antibiotic. On day 1st and day 7th after therapy, patients will be observed the presence of infection, inflammation, and increasing of eye pressure. On day 7th, day 30th, and day 90th after therapy, patients will do a visual field test, visual acuity test, electroretinography, funduscopy, and Optical Coherence Tomography examination. The observation results will be written in the observation table and analyzed by a statistic. After the data was completed, then make a discussion, conclusion, and suggestion of the study.
- Conditions
- Retinitis Pigmentosa
- Being tested
- Umbilical Cord Mesenchymal Stem Cell (UC-MSC), Conditioned Medium (CM)
- Keywords
- Allogeneic Mesenchymal Stem Cell, Umbilical Cord Mesenchymal Stem Cell, Conditioned Medium, Allogeneic Umbilical Cord Mesenchymal Stem Cell
- Medical subject headings
- Retinitis Pigmentosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Mesenchymal Stem Cell (UC-MSC)Biological
Umbilical Cord Mesenchymal Stem Cell (UC-MSC) injected by peribulbar
Conditioned Medium (CM)Biological
Conditioned Medium (CM) injected by peribulbar
| Study group | Role | What they receive |
|---|---|---|
| Conditioned Medium (CM) | Active comparator | a total 2 ml volume of Conditioned Medium derived Umbilical Cord Mesenchymal Stem Cell will be injected by peribulbar Receives: Biological: Conditioned Medium (CM) |
| UC-MSC + NaCl | Active comparator | 1.8 ml cell preparations are suspended in physiological NaCl until it reaches a total of 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) suspension will be injected by peribulbar Receives: Biological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC) |
| UC-MSC+CM | Active comparator | 1.8 ml cell preparations are suspended in Conditioned Medium (CM) until it reaches total of 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) + Conditioned Medium (CM) suspension will be injected by peribulbar Receives: Biological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC); Biological: Conditioned Medium (CM) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual Acuity Test
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Measured: 1 week after injection
Visual Acuity Test
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Measured: 1 month after injection
Visual Acuity Test
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Measured: 3 months after injection
Visual Field Test
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Measured: 1 week after injection
Visual Field Test
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Measured: 1 month after injection
Visual Field Test
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Measured: 3 months after injection
Funduscopy
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Measured: 1 week after injection
Funduscopy
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Measured: 1 month after injection
Funduscopy
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Measured: 3 months after injection
Electrorectinography
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Measured: 1 week after injection
Electrorectinography
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Measured: 1 month after injection
Electrorectinography
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Measured: 3 months after injection
Optical Coherence Tomography (OCT)
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Measured: 1 week after injection
Optical Coherence Tomography (OCT)
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Measured: 1 month after injection
Optical Coherence Tomography (OCT)
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Measured: 3 months after injection
Angiography
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Measured: 1 week after injection
Angiography
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Measured: 1 month after injection
Angiography
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Measured: 3 months after injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2020
- Study start7 Oct 2018
- Primary completion20 Jun 2019
- Study completion20 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis PigmentosaStudies of the same condition, open and under way.
- More trials run by PT. Prodia Stem Cell IndonesiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- PT. Prodia Stem Cell Indonesia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- dr Cosmos O Mangunsong, Sp.M, Principal investigator, Gadjah Mada University, Faculty of MedicineProfessor dr. Yohanes W Wirohadidjojo, Sp.KK(K), PhD, Study director, Gadjah Mada University, Faculty of MedicineBayu W Putera, S.Si, M.Kes, Study director, Prodia Stem Cell Indonesiadr Bayu M Sasongko, Sp.M, PhD, Study director, Gadjah Mada University, Faculty of Medicinedr Melita S Djaja, Sp.M, Study chair, Jakarta Eye Centerdr Amyra D Costa, Study chair, Jakarta Eye CenterRima Haifa, B.Sc, Study chair, Prodia StemCell Indonesia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04315025
- Sponsor's protocol ID
- CT/RP/02/2018
- Acronym
- RP
- Status last verified
- Mar 2020
- First submitted
- 8 Mar 2020
- Last updated
- 4 Jun 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety Issues of Peribulbar Injection of Umbilical Cord Mesenchymal Stem Cell (UC-MSC) in Patients With Retinitis Pigmentosa
- ClinicalTrials.gov ID
- NCT04315025
- Acronym / sponsor ID
- RP / CT/RP/02/2018
- Conditions
- Retinitis Pigmentosa
- Keywords
- Allogeneic Mesenchymal Stem Cell, Umbilical Cord Mesenchymal Stem Cell, Conditioned Medium, Allogeneic Umbilical Cord Mesenchymal Stem Cell
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- PT. Prodia Stem Cell IndonesiaIndustry (pharma, biotech, devices)
- Study start
- 7 Oct 2018 (actual)
- Primary completion
- 20 Jun 2019 (actual)
- Study completion
- 20 Sep 2019 (actual)
- First posted
- 19 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study will perform UC-MSCs and CM transplantation. The first group will be injected by UCMSC+NaCl. the 2nd group will be injected by UC-MSC+CM. the 3rd group will be injected by CM. Each group consists of 6 subjects. all groups will be transplanted via peribulbar route. the dosage of UC-MSC is 1 million cells for each subject. All groups will be observed until 6 months.
Detailed description
The eyes which will give the transplant should be given an aseptic and antiseptic technique to prevent the contamination from the inside and outside. Sterile cover attached to other parts of the face except for the eyes. 1.8 ml cell preparations are suspended in physiological NaCl until it reaches a total of 2 ml volume of cell suspension (for UC-MSC + NaCl group). Stem cell suspension will be injected by peribulbar and if the injection was done, patients will be given a quinolone antibiotic. On day 1st and day 7th after therapy, patients will be observed the presence of infection, inflammation, and increasing of eye pressure. On day 7th, day 30th, and day 90th after therapy, patients will do a visual field test, visual acuity test, electroretinography, funduscopy, and Optical Coherence Tomography examination. The observation results will be written in the observation table and analyzed by a statistic. After the data was completed, then make a discussion, conclusion, and suggestion of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Conditioned Medium (CM) | Active comparator | Biological: Conditioned Medium (CM)a total 2 ml volume of Conditioned Medium derived Umbilical Cord Mesenchymal Stem Cell will be injected by peribulbar |
| UC-MSC + NaCl | Active comparator | Biological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC)1.8 ml cell preparations are suspended in physiological NaCl until it reaches a total of 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) suspension will be injected by peribulbar |
| UC-MSC+CM | Active comparator | Biological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC), Biological: Conditioned Medium (CM)1.8 ml cell preparations are suspended in Conditioned Medium (CM) until it reaches total of 2 ml volume of cell suspension. Umbilical Cord Mesenchymal Stem Cell (UC-MSC) + Conditioned Medium (CM) suspension will be injected by peribulbar |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Visual field defects at initial examination with Humhprey perimetry are between 25% to 50%
- Willing to sign informed consent as research subjects
- Willing to do peribulbar injection with mesenchymal stem cells isolated from umbilical cord tissue
- Willing to do visual field checks with Humphrey's perimetry, vision tests with Snellen boards, Optical Coherrent Tomography (OCT) examinations, electroretinogram examinations and fill out a quality of life questionnaire
Key exclusion criteria (6 listed)
- Pregnant or nursing women
- Positive result of HIV test
- Have a history of eye tumors
- In immunosuppressive treatment or other drugs that can affect the growth of transplanted stem cells
- Have another eye disease such as diabetic retinopathy, uveitis, cataract, and glaucoma
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord Mesenchymal Stem Cell (UC-MSC)Umbilical Cord Mesenchymal Stem Cell (UC-MSC) injected by peribulbar | Biological | UC-MSC + NaCl, UC-MSC+CM |
| Conditioned Medium (CM)Conditioned Medium (CM) injected by peribulbar | Biological | Conditioned Medium (CM), UC-MSC+CM |
Primary outcome
Visual Acuity Test
Time frame: 1 week after injection
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Visual Acuity Test
Time frame: 1 month after injection
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Visual Acuity Test
Time frame: 3 months after injection
a test to see the sharpness of vision using Snellen Chart which is read at a distance of 6 meters. Normal eye test results are stated in 6/6.
Visual Field Test
Time frame: 1 week after injection
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Visual Field Test
Time frame: 1 month after injection
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Visual Field Test
Time frame: 3 months after injection
a test to assess the breadth of views of the lateral, medial, superior and inferior aspects using Humprey's perimetry.
Funduscopy
Time frame: 1 week after injection
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Funduscopy
Time frame: 1 month after injection
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Funduscopy
Time frame: 3 months after injection
an examination to see and assess the abnormalities and conditions in the ocular fundus using an ophthalmoscope. This examination is carried out by an ophthalmologist.
Electrorectinography
Time frame: 1 week after injection
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Electrorectinography
Time frame: 1 month after injection
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Electrorectinography
Time frame: 3 months after injection
an examination to provide a picture of objectivity, a quantitative measure of retinal function to monitor the function of stem cells and ganglion cells in the eye. This examination is carried out by a trained ophthalmologist.
Optical Coherence Tomography (OCT)
Time frame: 1 week after injection
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Optical Coherence Tomography (OCT)
Time frame: 1 month after injection
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Optical Coherence Tomography (OCT)
Time frame: 3 months after injection
is a diagnostic test that give the information about posterior segment structure, which is retina and optic nerve papillae so that it can be used to assess macular abnormalities
Angiography
Time frame: 1 week after injection
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Angiography
Time frame: 1 month after injection
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Angiography
Time frame: 3 months after injection
a process of taking photos of the retina by injecting fluorescent to get a bleeding location on the retina.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2020.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
dr Cosmos O Mangunsong, Sp.M principal investigator
Gadjah Mada University, Faculty of Medicine
Professor dr. Yohanes W Wirohadidjojo, Sp.KK(K), PhD study director
Gadjah Mada University, Faculty of Medicine
Bayu W Putera, S.Si, M.Kes study director
Prodia Stem Cell Indonesia
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Umbilical Cord Mesenchymal Stem Cell (UC-MSC) and Conditioned Medium (CM) in people with Retinitis Pigmentosa, run by PT. Prodia Stem Cell Indonesia at 2 sites in Indonesia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Indonesia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Visual Acuity Test (time frame: 1 week after injection). The study lists 18 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2020.